Addressing Unmet Medical Needs in Autism Spectrum Disorder

According to the Centers for Disease Control and Prevention (CDC), approximately 1 in 36 children under the age of 8 may be diagnosed with Autism Spectrum Disorder (ASD). Core symptoms of ASD include impairments in social interaction, communication, and the presence of stereotyped repetitive behaviors. Common co-occurring conditions that impact quality of life are extensive and diverse, and include irritability, anxiety, ADHD, allergies, autoimmune disorder, neuroinflammation, and epilepsy. Physicians have reported that irritability impacts a majority of pediatric ASD patients. The presentation of ASD-associated irritability can vary with severity and age and can be caused by a broad array of different factors including lack of sleep, the inability to communicate pain, and mental health disorders. It manifests in a range of behaviors including aggressive behavior, self-harming, isolation, and emotional meltdowns. Limited treatment options currently exist and those that are approved can have significant side effects.

A significant area of unmet medical need for children with ASD is a treatment to address the core symptoms and co-occurring conditions associated with ASD such as irritability. The prevalence and severity of irritability and anxiety challenges faced by people with autism and the need of safe and effective treatment options make ASD an important focus of novel drug discovery to bring forward innovative medical interventions.

Axial is developing AB-2004, a therapeutic candidate for the reduction of irritability in children with autism.

Axial’s novel therapeutic approach is focused on the development of small molecule therapeutics with defined mechanisms of action against new targets in the gut to mitigate the impact of metabolites and bacteria linked to the pathology, progression, and symptoms of disease, with an initial focus on neurological diseases and disorders.

Clinical Trial

Axial’s lead product candidate, AB-2004, is a gut-restricted molecular therapy being studied for the treatment of irritability associated with autism in a Phase 2b trial.

For more information about the clinical trial, vistit


Harnessing the Connection Between the Gut and the Brain

AB-2004 is Axial Therapeutic’s lead investigational therapy that targets the microbiome gut-brain axis and its role in co-occurring conditions associated with Autism Spectrum Disorder (ASD). AB-2004 is a gut-targeted, molecular therapeutic, which means it is designed to work in the gut only and does not enter other bodily tissues. We believe this mechanism may result in minimal side effects due to lack of exposure across other parts of the body, something we are verifying in controlled clinical trials.

Irritability in Autism and a New Therapeutic Approach

Irritability is a common co-occurring condition in children with autism, presents with varying severity and manifests in a range of behaviors including aggressive behavior, self-harming, isolation, and emotional meltdowns. Limited treatment options currently exist to address irritability associated with autism and those that are approved can have significant side effects.

Scientific evidence has shown there may be a link between bacteria commonly found in the digestive tract and the brain that could contribute to certain challenging behaviors, such as irritability and anxiety, AB-2004 is being studied as a potential new treatment for managing irritability associated with ASD in children. AB-2004 is designed to adsorb certain substances produced by gut bacteria to reduce their ability to enter the bloodstream and reach the brain.

AB-2004, An Innovative Product Designed to Improve Lives

The active ingredient in AB-2004 is a highly engineered form of spherical carbon designed with human safety and biological selectivity in mind, making it very different from activated charcoal. Each sphere of AB-2004 consists of a network of pores that allows it to selectively adsorb metabolites that may contribute to characteristics associated with ASD like irritability and anxiety.

AB-2004 is a tasteless and odorless powder formulation that can be mixed with soft food which makes taking the medication very easy. Finally, AB-2004 will not discolor your hands or your food.

AB-2004 has several critical attributes that make it potentially ideal for chronic clinical use and differentiate it from activated charcoal available over the counter.

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